Price for Spark's gene therapy for blindness set at $850,000

The wholesale acquisition cost of one-time Luxturna treatment for confirmed biallelic RPE65 mutation-associated retinal dystrophy is $425,000 per eye, Spark Therapeutics confirmed in an email to Healio.com/OSN.

Perspective from Julia A. Haller, MD

Spark Therapeutics also announced three new payer programs: an outcomes-based rebate arrangement with long-term durability measure, a contracting model and a proposal to CMS under which payments for Luxturna (voretigene neparvovec-rzyl) would be made over time, according to a company release.

Luxturna will cost $850,000 total to treat both eyes. Biallelic RPE65 mutation-associated retinal dystrophy affects 1,000 to 2,000 patients in the U.S., according to the FDA.

In its outcomes-based rebate arrangement, Spark Therapeutics reported it will share risk with certain health insurers and pay rebates if patient outcomes fail to meet specified thresholds.

For its contract model, Spark Therapeutics would enter into an agreement with commercial payers under which the payer or payer’s specialty pharmacy purchases Luxturna. The payer agrees to provide coverage for its members consistent with FDA labeling of Luxturna, expedite benefits processing and cap patient out-of-pocket amounts at in-network limits. Additionally, the payer would agree with the treatment center on reimbursement, according to a company release.

Finally, Spark Therapeutics is seeking to allow customers to pay for Luxturna in installments. However, under current government price reporting requirements, Spark Therapeutics cannot offer installment payments or outcomes-based rebates above a certain threshold. The company has submitted a proposal to CMS to enable commercial and government payers an installment option and greater rebates tied to clinical outcomes, according to a release.

The company has reached an agreement in principle with Harvard Pilgrim to make the therapy available under the outcomes-based rebate arrangement and contracting model. It also reached an agreement in principle with affiliates of Express Scripts for the contracting model, according to the company. – by Robert Linnehan

Perspective

Julia A. Haller, MD

On the one hand, what is the price for vision? We are talking about kids going blind, and now they can see. At the FDA hearing during the public comments, I was tearing up listening to these children who couldn’t see at all when it started to get dark and couldn’t play with their friends at night. They were bullied and thought to be stupid because they couldn’t see. These poor children who led such sadly constricted lives, and then they were transformed. It was a dramatic reversal of this blinding eye disease. What is that worth? Can you compare it to treatment for cancer or other serious diseases that cost hundreds of thousands of dollars and justify it? I don’t know the answer to that. Also, having been a small part of the study and seeing how long it took to develop this groundbreaking and transformational therapy, which is ushering in a whole new era, and to see how much time, effort, creativity, persistence and funding it took to this point, what is the right reimbursement for that? It is not like building a car. This took decades. I see why the price tag would be high. On the other hand, there are so many people who are going to need gene therapy, and how can we possibly treat everyone at that price? You can see the company trying to figure out some novel ways to make this work, and I give them a lot of credit. These are out-of-the-box ideas — the money-back guarantee, for example. Also, trying to spread out the cost if you change insurance plans so the original plan would not be stuck with the whole cost if you migrate to another one. They are trying to grapple with the practical and ethical issues of this. I was impressed to see all of the thought going into it and intrigued to read about it. I am hopeful that some of these models will be workable because we are going to need different models if we are going to cure all the genetic eye diseases we have out there.

Julia A. Haller, MD

OSN Retina/Vitreous Board Member

Disclosures: Haller reports she was a surgeon on the Luxturna FDA clinical trial and she was reimbursed for her time and travel to the FDA presentation by Spark Therapeutics.